RNA-binding plus ribosomal proteins block c-Myc in lung cancer
Lung adenocarcinoma may be cornered by a recent finding coming from a study published on Nov. 30, 2023, in the Proceedings of the National Academy of Sciences. This investigation has unveiled that a tumor suppressor molecule called RNA-binding motif protein 10 (RBM10) partners with two ribosomal proteins, which in turn inactivate the well-known oncogene Myc proto-oncogene protein (c-Myc).

RNA-binding plus ribosomal proteins block c-Myc in lung cancer
Lung adenocarcinoma may be cornered by a recent finding coming from a study published on Nov. 30, 2023, in the Proceedings of the National Academy of Sciences. This investigation has unveiled that a tumor suppressor molecule called RNA-binding motif protein 10 (RBM10) partners with two ribosomal proteins, which in turn inactivate the well-known oncogene Myc proto-oncogene protein (c-Myc).
DCZ-5417 suppresses multiple myeloma progression, study shows
Norcantharidin is an active ingredient in Chinese traditional medicine that has activity against several cancer types, but its application is limited in the clinic due to toxicity and a narrow treatment window. Researchers from Tongji University and the Shanghai Institute of Materia Medica have recently described a derivative of norcantharidin – DCZ-5417 – for the potential treatment of multiple myeloma (MM).
Cereno completes preclinical safety program for antithrombotic candidate
Cereno Scientific AB has completed the preclinical safety program for CS-014, a histone deacetylase (HDAC) inhibitor in development for arterial and venous thrombosis prevention.
Ebviously presents in vitro data on EBV vaccine candidate
Ebviously GmbH, a spin-off from Helmholtz Munich (HMGU), has presented new in vitro data for its Epstein-Barr virus (EBV) vaccine candidate EBV-001. Based on noninfectious virus-like particles (VLPs) derived from EBV, EBV-001 is designed as a highly immunogenic, multiantigen vaccine to prevent EBV-associated diseases, such as infectious mononucleosis.
Character Biosciences patents new polypeptides for AMD
A Character Biosciences Inc. patent describes polypeptides acting as low-density lipoprotein receptor (LDLR) ligands and reported to be useful for the treatment of age-related macular degeneration (AMD).
Iama Therapeutics’ IAMA-6 shows promise in preclinical mesial temporal lobe epilepsy
Intracellular chloride levels are crucial for physiological brain development and correct functioning. On the other hand, aberrant expression of chloride importer NKCC1 (basolateral Na-K-Cl symporter, SLC12A2) and exporter KCC2 (K-Cl cotransporter 2, SLC12A5) is involved in neurodevelopmental and neurologic conditions, including mesial temporal lobe epilepsy (MTLE), among others, leading to an imbalance between excitatory and inhibitory signals.
Psychogenics licenses rights to ENT1 inhibitor for neuropsychiatric disorders
PGI Drug Discovery LLC, the drug discovery arm of Psychogenics Inc., has entered into an exclusive license agreement with F. Hoffmann-La Roche Ltd. to obtain global development, manufacturing and commercialization rights to RO-7117997. Psychogenics intends to evaluate RO-7117997 as a potential treatment for several psychiatric and neurological conditions.
New NLRP3 inflammasome inhibitors reported in Ventus Therapeutics patent
Ventus Therapeutics US Inc. has disclosed oxoindolinyl amide derivatives acting as NLRP3 inflammasome inhibitors. As such, they are reported to be useful for the treatment of cancer, metabolic, autoimmune diseases, liver, renal, respiratory, cardiovascular and inflammatory disorders, among others.

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Conferences

Bifunctional molecule capsid modulator and cGAS agonist achieves complete cure in AAV-HBV mice
American Association for the Study of Liver Diseases
LW-231, under development by Shanghai Longwood Biopharmaceuticals Co. Ltd., is a novel bifunctional molecule designed to simultaneously modulate hepatitis B virus (HBV) capsid assembly and activate cGAS-STING pathway. Preclinical data were recently presented.
Stem, immune and nervous cells all forces for tumor progression
Cancer
One of the difficulties for preventing the evolution of a tumor is that cancer progression can be promoted by undifferentiated or migrating cells whose states could follow different directions. At the 40th edition of Barcelona Biomed conferences at the Institute for Research in Biomedicine (IRB...
New Alzheimer's disease model includes cholinergic dysfunction
Neurology/psychiatric
Researchers from Nagoya City University presented the development of a new mouse model with cholinergic dysfunction and amyloid pathogenesis for Alzheimer's disease.
New pemphigus vulgaris mouse model better mimics human disorder
Dermatologic
Pemphigus vulgaris is an autoimmune skin disease mediated by autoantibodies to desmoglein-3 (DSG3). Since the adult murine model of this disease is quite limited, researchers aimed to develop a novel active adult pemphigus vulgaris murine model.

Cancer

China Pharmaceutical University reports discovery of novel EGFR triple-mutation inhibitors
Most patients with non-small-cell lung cancer (NSCLC) develop acquired drug resistance to EGFR inhibitors. Osimertinib, a third-generation inhibitor, often sees its therapeutic effect reduced due to the C797S mutation of EGFR exon 20, which blocks the binding of Cys797 to osimertinib.

Infection

Dual Mcl-1 and Bcl-2 inhibition as promising approach for treating tuberculosis
Tuberculosis still kills a lot of people worldwide (1.6 million deaths per year). Previous findings demonstrated that induced myeloid leukemia cell differentiation protein Mcl-1 inhibitors reduced the growth of Mycobacterium tuberculosis in human macrophages, but when inhibiting multiple proteins...

New dual inhibitors of host proteases TMPRSS2 and CTSL/CTSB as potential anti-SARS-CoV-2 agents
Coronavirus
Current antiviral agents for COVID-19 treatment target viral proteins, which are susceptible to mutation during SARS-CoV-2 evolution. A potential strategy to fight emerging drug resistances is the development of compounds targeting host proteins that are indispensable for the viral life cycle.
Ethris gains clearance for phase I study of ETH-47 for respiratory viral infections
Regulatory
Ethris GmbH has received approval from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to proceed with a first-in-human trial of its inhaled mRNA program, ETH-47, in healthy participants for the treatment and prophylaxis of respiratory viral infections.

Neurology/psychiatric

Nurexone reports new siRNA sequences with potential for spinal cord injury
RNA
Nurexone Biologic Inc. has reported results from laboratory tests of its secondary two proprietary sequences, showing promise for the treatment of spinal cord injuries.
Brain penetration profile of Nodthera’s NLRP3 inflammasome inhibitor described
Inflammatory
Aberrant NLRP3 inflammasome activation is at the root of a wide number of conditions, from heart, gastrointestinal, kidney or liver disorders to neuroinflammatory diseases such as Parkinson’s disease or multiple sclerosis. Researchers from Nodthera Ltd. have reported on NT-0796, an NLRP3...
miR-92a confers protection against inflammation-driven neurodegeneration
Biomarkers
MicroRNAs (miRNAs) play an important role in modulating neuronal stress responses, but their impact on neuroprotection has been poorly studied. Multiple sclerosis (MS) is the most common inflammatory disease affecting the central nervous system. Researchers have recently attempted to identify...
Shanghai Institute of Organic Chemistry discovers new KEAP1/NRF2/ARE pathway activators
Patents
Shanghai Institute of Organic Chemistry has described oleanamide derivatives acting as Kelch-like ECH-associated protein 1 (KEAP1)/nuclear factor erythroid 2-related factor 2 (NFE2-related factor 2; NFE2L2; NRF2)/antioxidant response element (ARE) pathway activators reported to be useful for the...
DE Shaw Research describes new TRPA1 antagonists
Patents
DE Shaw Research LLC has identified transient receptor potential cation channel subfamily A member 1 (TRPA1) antagonists reported to be useful for the treatment of pain, fibrosis, autoimmune disease, neurological, dermatological, cardiovascular, respiratory and gastrointestinal disorders, among...

Endocrine/metabolic

FDA clearance for first-in-human study of Uniqure’s AMT-191 for Fabry disease
Regulatory
Uniqure NV has received FDA clearance of its IND application for AMT-191, the company’s gene therapy candidate for Fabry disease.

Biomarkers

Somatic Ras-MAPK activating variants linked to mesial temporal lobe epilepsy
Conferences
Researchers from The Brigham and Women's Hospital presented data from a study that aimed to evaluate the role of somatic variants in drug-resistant mesial temporal lobe epilepsy (MTLE). High-coverage whole-exome sequencing was conducted using DNA samples derived from the hippocampus and paired...

Researchers find de novo CAMK2B variant in patient with tetralogy of Fallot
Conferences
CAMK2B encodes calcium/calmodulin-dependent protein kinase type II subunit β, a kinase expressed in the brain that has an important role in synaptic plasticity. Genetic variations are associated with neurodevelopmental disorders.
Researchers find KAT6B is deleterious variant behind neuroblastoma
Conferences
The GOBACK study analyzed germline susceptibility in children with birth defects and a cancer diagnosis, including a total of 47 probands. Whole-genome sequencing was performed, and researchers evaluated single-nucleotide variants, insertion/deletion variants, as well as copy number variations.

Gastrointestinal

Esperion Therapeutics discovers new ACLY inhibitors
Patents
Esperion Therapeutics Inc. has described ATP citrate lyase (ACLY) inhibitors reported to be useful for the treatment of cancer and more.
ZHX2 promotes liver recovery after acute liver injury
Dysfunction in the mitochondria contributes to the development of acute liver injury (ALI). There is emerging evidence indicating the mitochondria is key to maintain liver homeostasis and survival; thus, controlling its functioning is important...
Novel hDHODH inhibitor for inflammatory bowel disease described
Human dihydroorotate dehydrogenase (hDHODH) is a mitochondrial enzyme participating in de novo pyrimidine biosynthesis essential for T- and B-lymphocyte proliferation.
Investigators find lactic acid bacteria strain that alleviates IBS in rats
Irritable bowel syndrome (IBS) is a functional gastrointestinal disorder where visceral hypersensitivity (VH), which causes discomfort and negatively impacts the quality of life, is influenced by stressful events. In recent years, the use of...
Lerna Biopharma presents first-in-class GalNAc-siRNA therapeutic for treatment of liver diseases
Conferences
Researchers from Lerna Biopharma Pte. Ltd. (formerly Cargene Therapeutics Pte. Ltd.) recently presented the discovery and preclinical evaluation of a first-in-class GalNAc-siRNA therapeutic, CG-LR1, being developed for the treatment of liver...
CVI Pharmaceuticals CVI-2742 shows promise for treating NASH
Conferences
CVI Pharmaceuticals has presented preclinical data on their thyroid hormone receptor beta (THRB) selective agonist CVI-2742 for the potential treatment of NASH. Selective activation of the THRB contributes to ameliorating the symptoms of...

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